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Hematopoietic Stem Cell Gene Transfer for Hemophilia A
NCT03818763Phase 1Active, not recruiting
- Sponsor
- Medical College of Wisconsin
- Start
- Apr 2020
- Primary completion
- Mar 2027
- Countries
- 1
01
Overview
Phase 1, open-label, single-arm trial of autologous CD34+ peripheral blood stem cells expressing B-domain deleted Factor VIII in hemophilia A, sponsored by Medical College of Wisconsin. Primary endpoint: Number of enrolled participants with adequate gene transduced hematopoietic stem cells for FVIII gene therapy infusion at 4 years. Started Apr 2020; status: active, not recruiting.
02
Drugs
autologous CD34+ peripheral blood stem cells expressing B-domain deleted Factor VIII
03
Conditions
hemophilia A
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